Key result
Lipid-lowering therapy was effective with low discontinuation-related adverse events in children with HeFH, though achieving LDL-C treatment goals was relatively rare.
Why the study?
Does lipid-lowering therapy (LLT) improve achievement of low density lipoprotein cholesterol (LDL-C) goals in children with heterozygous familial hypercholesterolaemia (HeFH)?
Meta-Analysis
Yes
Does lipid-lowering therapy (LLT) improve achievement of low density lipoprotein cholesterol (LDL-C) goals in children with heterozygous familial hypercholesterolaemia (HeFH)?
In children with HeFH, lipid-lowering therapy is efficacious and safe, but achieving LDL-C goals remains rare, highlighting the need for early combination therapy.
Aims: Heterozygous familial hypercholesterolaemia (HeFH) is one of the most frequent monogenic disorders in the world, leading to premature atherosclerotic cardiovascular diseases. The aim of this meta-analysis was to evaluate the efficacy and safety of lipid-lowering therapy (LLT) and achievement of low density lipoprotein cholesterol (LDL-C) goal in children with HeFH. Conclusion: Despite the efficacy of LLT in children with HeFH and the low occurrence of discontinuation-related adverse events, achieving LDL-C treatment goals was relatively rare, with large differences between the investigated countries. These results underscore the importance of considering early combination therapy of statins and ezetimibe, and PCSK9 inhibitors (if available) to attain LDL-C goals effectively.
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Bytyçi et al. (2025) conducted a meta-analysis in Heterozygous familial hypercholesterolaemia (HeFH). Lipid-lowering therapy (LLT) was evaluated on Efficacy and safety of lipid-lowering therapy and achievement of LDL-C goal. Lipid-lowering therapy was effective with low discontinuation-related adverse events in children with HeFH, though achieving LDL-C treatment goals was relatively rare.
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